-Key features of the REGENERATE Phase 3 trial in non-cirrhotic NASH with liver fibrosis finalized pursuant to consultations with FDA and EMA
-REGENERATE trial design intended to support initial approval on interim 72-week histology endpoint and full approval on liver-related outcomes endpoint
-Conference Call Scheduled Tuesday, May 19 at 8:00 a.m. ET
NEW YORK, May 19, 2015 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept) today announced its plans for an international Phase 3 trial of obeticholic acid (OCA), the company's lead FXR agonist, in patients with non-cirrhotic nonalcoholic steatohepatitis (NASH) with liver fibrosis. OCA has received breakthrough therapy designation in this patient population from the U.S. Food and Drug Administration (FDA). In accordance with harmonized advice from the FDA and European Medicines Authority (EMA), the Randomized Global Phase 3 Trial to Evaluate the Impact on NASH with Fibrosis of Obeticholic Acid Treatment (REGENERATE) has been designed as a double-blind, placebo-controlled pivotal Phase 3 clinical trial expected to enroll up to approximately 2,500 patients and assess the potential benefit of OCA treatment on liver-related clinical outcomes. The trial will include a pre-planned interim histology analysis after 72 weeks of treatment in approximately 1,400 patients which is intended to serve as the basis for seeking U.S. and international marketing approvals of OCA for the treatment of NASH patients with liver fibrosis. Intercept will hold a conference call and audio webcast today at 8:00 a.m. ET to discuss aspects of the planned Phase 3 trial. Conference call details are provided below.
The REGENERATE trial will be conducted at approximately 250 centers in North America, Europe and other regions and trial initiation is anticipated in 3Q 2015. Patients will be randomized 1:1:1 to one of placebo, 10 mg of OCA, or 25 mg of OCA, taken once daily. The study population will primarily be comprised of NASH patients with stage 2 or stage 3 advanced liver fibrosis who will be evaluated on the primary efficacy endpoints in the 72-week interim analysis and subsequently. In addition, a relatively small group of NASH patients with stage 1 early liver fibrosis with an increased risk of rapid progression due to concomitant diabetes, obesity or active liver inflammation indicated by elevated ALT will also be enrolled, but not included in the primary endpoint analyses.
The interim analysis after 72 weeks of treatment in approximately 1,400 patients will be conducted on the following co-primary endpoints, which are intended to serve as the basis for seeking U.S. and international marketing approvals: (i) the proportion of OCA-treated patients relative to placebo achieving at least one stage of liver fibrosis improvement with no worsening NASH; and (ii) the proportion of OCA-treated patients relative to placebo achieving NASH resolution with no worsening of liver fibrosis. Additional supportive histologic endpoints and non-invasive markers of liver fibrosis and steatohepatitis will also be evaluated. The REGENERATE trial will remain blinded after the interim analysis and continue to follow patients until the occurrence of a pre-specified number of adverse liver-related clinical events, including progression to cirrhosis, to confirm clinical benefit on a post-marketing basis.
"We are very pleased to announce the key aspects of our planned Phase 3 REGENERATE trial today, a culmination of our analyses of the FLINT trial results in OCA-treated NASH patients with advanced liver fibrosis and extensive collaboration with U.S. and European regulatory agencies," said Mark Pruzanski, M.D., President and Chief Executive Officer. "There are no approved medicines available to treat the disease which is why NASH is predicted to become the leading indication for liver transplant within the next few years."
"Recently presented data analyses in patients with NASH and liver fibrosis from the FLINT trial suggest that in addition to OCA's apparent ability to resolve NASH, OCA appears to meaningfully improve liver fibrosis, particularly in those patients at higher risk of progressing to cirrhosis," said Vlad Ratziu, M.D., Ph.D., Professor of Hepatology at the Pierre and Marie Curie University, Paris. "A benefit on liver fibrosis is important since studies have consistently shown it to be the best histological predictor of both liver-related complications and all-cause mortality."
Conference Call on May 19 at 8:00 a.m. ET
Intercept will hold a conference call and webcast on Tuesday, May 19 at 8:00 a.m. ET. The live event will be available on the investor page of the Intercept website at http://ir.interceptpharma.com or by calling (855) 232-3919 (toll-free domestic) or (315) 625-6894 (international) five minutes prior to the start time (no passcode is required). A replay of the call will be available on the Intercept website approximately two hours after the completion of the call and will be archived for two weeks.
About Intercept
Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat neglected chronic liver diseases. The company's lead product candidate, obeticholic acid (OCA), is an agonist of the farnesoid X receptor (FXR). OCA is being developed for a variety of chronic liver diseases, including primary biliary cirrhosis (PBC), nonalcoholic steatohepatitis (NASH), primary sclerosing cholangitis (PSC) and biliary atresia. The FDA has granted OCA breakthrough therapy designation for the treatment of NASH with liver fibrosis, a population representing potentially more than 14 million patients in the United States, and granted OCA fast track designation for the treatment of patients with PBC who have an inadequate response to or are intolerant of ursodiol. OCA has also received orphan drug designation in both the United States and Europe for the treatment of PBC and PSC. Intercept owns worldwide rights to OCA outside of Japan, China and Korea, where it has out-licensed the product candidate to Sumitomo Dainippon Pharma. For more information about Intercept, please visit the Company's website at: www.interceptpharma.com.
Safe Harbor Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the clinical, preclinical and regulatory developments for our product candidates, including the design and powering of the Phase 3 clinical trial of OCA in NASH with liver fibrosis, the anticipated results of our clinical and preclinical trials and other development activities and the timing thereof, our plans and ability to seek marketing approval for OCA in NASH with liver fibrosis on the basis of a pre-planned interim analysis, our potential development and regulatory milestones and the timeframes under which we anticipate such milestones may be achieved, including the anticipated initiation of the Phase 3 clinical trial of OCA in NASH with liver fibrosis, our plans in relation to other subpopulations of patients with NASH, and our strategic directives under the caption "About Intercept." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of important risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: the initiation, cost, timing, progress and results of our development activities, preclinical studies and clinical trials; the timing of and our ability to obtain and maintain regulatory approval of OCA and any other product candidates we may develop, particularly the possibility that regulatory authorities may require clinical outcomes data (and not just results based on achievement of a surrogate endpoint) as a condition to any marketing approval for OCA, and any related restrictions, limitations, and/or warnings in the label of any approved product candidates; our plans to research, develop and commercialize our product candidates; the election by our collaborators to pursue research, development and commercialization activities; our ability to attract collaborators with development, regulatory and commercialization expertise; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to successfully commercialize our product candidates; the size and growth of the markets for our product candidates and our ability to serve those markets; the rate and degree of market acceptance of any future products; the success of competing drugs that are or become available; regulatory developments in the United States and other countries; the performance of third-party suppliers and manufacturers; our need for and ability to obtain additional financing; our estimates regarding expenses, future revenues and capital requirements and the accuracy thereof; our ability to retain key scientific or management personnel; and other factors discussed under the heading "Risk Factors" contained in our annual report on Form 10-K for the year ended December 31, 2014 filed on March 2, 2015 as well as any updates to these risk factors filed from time to time in our other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intercept undertakes no duty to update this information unless required by law.